Table 2.
THERAPEUTIC AGENT | MOLECULAR TARGET | CELLULAR TARGET | CLINICAL USE |
---|---|---|---|
MMF | IMPDH | T and B cells | AICs, many (25, 26) |
Rituximab | CD20 | mature B cells | AICs, many (27–30) |
Bortezomib | proteasome | plasma cells | AICs, MM (31–38) |
Daratumumab | CD38 | plasma cells and plasma blasts | refractory AICs, MM (39–43) |
Isatuximab | CD38 | Plasma cells and plasma blasts | refractory AICs, MM (44, 45) |
Belimumab | BAFF | B cells | AIC in SLE (46, 47) |
Atacicept | BAFF and APRIL | B cells | SLE (48) |
Ibrutinib | BTK | B cells | AIC in ALL (49, 50) |
Epratuzumab | CD22 | B cells | SLE (51) |
Sirolimus/Everolimus | mTOR | T cells | ALPS, CTLA4 def, APDS, others (52–55) |
Abatacept | CTLA4 | T cells | CTLA4 def (56, 57), LRBA def (58) |
Leniolisib | p110delta | T cells | APDS1 (59) |
Seletalisib | p110delta | T cells | APDS1 and APDS2 (60) |
Tofacitinib | JAK1, JAK3 | T cells and inflammasome | JAK/STAT diseases (61–64) |
Ruxolitinib | JAK1, JAK2 | T cells and inflammasome | SAVI, STAT1 GOF, STAT3 GOF (61, 62) |
Baricitinib | JAK1, JAK2 | T cells and inflammasome | JAK/STAT diseases (61–64) |
Filgotinib | JAK1 | T cells and inflammasome | JAK/STAT diseases (61–64) |
Decernotinib | JAK3 | T cells and inflammasome | JAK/STAT diseases (61–64) |
Anakinra | IL-1R | T and B cells | WAS (65), MIS-C (66, 67) |
Etanercept | TNF | macrophages | DADA2 (68) |
Infliximab | TNF | macrophages | DADA2 (68) |
Adalimumab | TNF | macrophages | DADA2 (68) |
Plexirafor | CXCR4 | neutrophils | WHIM (69, 70) |
Mavorixafor | CXCR4 | neutrophils | WHIM (71) |
Eculizumab | C5 | complement | Rescue therapy of refractory AIHA (72, 73) |
AIC, autoimmune cytopenia; ALL, acute lymphatic leukaemia; ALPS, autoimmune lymphoproliferative syndrome; APDS, Activated PI3K-kinase Delta Syndrome; DADA2, deficiency od adenosine deaminase; MIS-C, multisystem inflammatory syndrome in children; MM, multiple myeloma; MMF, mycophenolate mophetile; SAVI, STING-associated vasculopathy with onset in infancy; SLE, systemic lupus erythematosus; TNF, tumor necrosis factor; WHIM, Warts, hypogammaglobulinemia, infections, and myelokathexis; WAS, Wiskott-Aldrich Syndrome.